Idhifa (enasidenib)
An approved treatment for Acute Myeloid Leukemia.
The same compound appears under different names depending on the context. Here is how to identify Enasidenib wherever you encounter it, plus the key facts at a glance.
- Generic name
- Enasidenib
- Brand name
- Idhifa
- Development codes
- AG-221, CC-90007
- Drug class
- IDH2 inhibitor
- Manufacturer
- Bristol Myers Squibb
- How it's taken
- Oral tablet once daily
A once-daily pill for adults whose AML has relapsed or never responded and carries a mutation in the IDH2 gene, found in roughly 1 in 10 people with AML. Approved August 1, 2017 as the first IDH2 inhibitor; it works by making blasts mature rather than by killing them outright.
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Where Enasidenib fits
The only approved IDH2 inhibitor. IDH1 and IDH2 mutations are mutually exclusive in almost all cases, so the molecular report decides which drug applies. Trials combine enasidenib with azacitidine and venetoclax in newly diagnosed disease.
How Enasidenib works
A mutated IDH2 enzyme makes large amounts of a chemical called 2-hydroxyglutarate, which blocks the steps that let a young blood cell mature. Enasidenib blocks the mutant enzyme (R140 and R172 forms), 2-hydroxyglutarate falls, and the blasts start to differentiate into normal-looking blood cells[1]. Because the leukemia cells mature rather than die, responses can take months, and the maturing cells can trigger differentiation syndrome.
Mechanism: Oral inhibitor of mutant IDH2 that lowers 2-hydroxyglutarate and lets leukemia blasts mature
Side effects and safety
- Embryo-fetal toxicity. Can harm an unborn baby; contraception during treatment and for 2 months after (women) or 2 months after (men).
- Noninfectious leukocytosis. White cells can rise as blasts mature; hydroxyurea is used to bring the count down.
- Tumor lysis syndrome. In 6%; blood chemistry is checked before starting and at least every 2 weeks for 3 months.
- Raised bilirubin. Enasidenib interferes with bilirubin processing; a rise without liver enzyme changes is common and usually managed by dose adjustment.
- Blood counts and chemistry before starting and at least every 2 weeks for the first 3 months
- Watch for differentiation syndrome symptoms from the first days of treatment
- Pregnancy test before starting
Idhifa carries a boxed warning for differentiation syndrome, which affected 14% of trial patients and can be fatal; signs include fever, breathlessness, lung infiltrates, fluid around the lungs or heart, rapid weight gain, swollen lymph nodes, bone pain and kidney or liver problems, and it is treated with steroids and close monitoring[1].
The other warning is harm to an unborn baby[1]. The most common side effects (20% or more) were nausea (50%), diarrhea (43%), vomiting (34%), raised bilirubin and decreased appetite (34%); tumor lysis syndrome occurred in 6% and noninfectious leukocytosis (a rise in white cells from the maturing blasts) in 12%[1].
This is not a complete list of side effects. Talk to your doctor or pharmacist about what to expect and when to seek medical attention.
Taking Enasidenib
The dose is 100 mg by mouth once a day, with or without food, swallowed whole, until the leukemia progresses or side effects become unacceptable. Because responses are slow, the label recommends at least 6 months of treatment if the disease is not progressing[1]. A missed or vomited dose is taken as soon as possible the same day.
Availability and cost
Only available as the brand-name product.
Help paying for Idhifa
Pick your insurance to see which help fits. Drugmaker copay cards can't be used with Medicare, Medicaid or TRICARE; charity funds are the usual route there.
- Copay help
BMS Access Support Co-Pay Assistance Program for eligible commercially insured patients; Idhifa is listed on the BMS co-pay page and its terms appear after selecting the medicine. Not for government insurance.
For: private insurance · source - Free medicine program
Bristol Myers Squibb Patient Assistance Foundation provides free medication to eligible uninsured patients experiencing financial hardship. Call 800-736-0003 or visit bmspaf.org.
For: no insurance · source - Insurance and case manager help
BMS Access Support helps with benefits checks, prior authorizations and appeals and refers government-insured patients to independent charitable foundations. Monday to Friday, 8 AM to 8 PM ET.
For: private insurance, Medicare, Medicaid, no insurance, underinsured · source
Good to know: The Idhifa co-pay dollar cap was not readable without selecting the medicine on the BMS site.
Access and eligibility
Adults with relapsed or refractory AML and an IDH2 mutation detected by an FDA-approved test. It is not approved for newly diagnosed AML or for IDH1 mutations, which are treated with Tibsovo or Rezlidhia.
Access program details are provided for informational purposes and may vary based on insurance coverage, geographic location, and individual circumstances. Confirm current eligibility directly with the manufacturer or your specialty pharmacy.
How the targeted AML pills compare, by mutation
AML has 19 FDA-approved medicines, counting the 3 older chemotherapy drugs. This chart covers the 8 that target a specific gene change, because the result of your mutation test decides which of them could apply to you. Chemotherapy (7+3, Vyxeos), Venclexta, Mylotarg, Onureg, Daurismo and the APL drugs are described on the drug pages instead. Several of these drugs are only approved after relapse, and all of the FLT3, IDH and menin drugs can cause differentiation syndrome, so your leukemia doctor will weigh the mutation, your point in treatment and your heart and liver tests.
Acute Myeloid Leukemia has 8 FDA-approved treatments: Rydapt (midostaurin, approved 2017); Vanflyta (quizartinib, approved 2023); Xospata (gilteritinib, approved 2018); Tibsovo (ivosidenib, approved 2018); Rezlidhia (olutasidenib, approved 2022); Idhifa (enasidenib, approved 2017); Revuforj (revumenib, approved 2024); Komzifti (ziftomenib, approved 2025). Rydapt is typically used as newly diagnosed, with intensive chemotherapy.
| Drug | Mutation it targets | When it is approved | How it's taken | How often | Given with | FDA approved |
|---|---|---|---|---|---|---|
Rydapt midostaurin | FLT3 (ITD or TKD) | Newly diagnosed, with intensive chemotherapy | Oral capsule | Twice daily on days 8 to 21 of each chemotherapy cycle | 7+3 induction and high-dose cytarabine consolidation (hospital-based chemotherapy) | 2017 |
Vanflyta quizartinib | FLT3-ITD only | Newly diagnosed, with intensive chemotherapy, then maintenance | Oral tablet | Once daily, with chemotherapy and then up to 36 months of maintenance | 7+3 induction and cytarabine consolidation, then alone as maintenance (not after transplant) | 2023 |
Xospata gilteritinib | FLT3 (ITD or TKD) | Relapsed or refractory | Oral tablets | Once daily | Taken alone | 2018 |
Tibsovo ivosidenib | IDH1 | Newly diagnosed (older or unfit) and relapsed or refractory | Oral tablets | Once daily | With azacitidine or alone (newly diagnosed, 75+ or unfit); alone at relapse | 2018 |
Rezlidhia olutasidenib | IDH1 | Relapsed or refractory | Oral capsule | Twice daily on an empty stomach | Taken alone | 2022 |
Idhifa enasidenib You are here | IDH2 | Relapsed or refractory | Oral tablet | Once daily | Taken alone | 2017 |
Revuforj revumenib | KMT2A rearrangement (any acute leukemia) or NPM1 mutation (AML) | Relapsed or refractory (NPM1: after other options) | Oral tablets | Twice daily | Taken alone; adults and children 1 year and older | 2024 |
Komzifti ziftomenib | NPM1 mutation | Relapsed or refractory, after other options | Oral capsules | Once daily on an empty stomach | Taken alone; adults only | 2025 |
This chart is for education only and is not medical advice. Approval dates, indications and warnings come from each drug's FDA prescribing information and Drugs@FDA. None of these drugs has been compared head to head with another in the same column, and trial results are not comparable across different studies. A leukemia specialist who has your mutation report will know which, if any, applies to you and whether a clinical trial would fit better.
Clinical trial results
Study AG221-C-001 (NCT01915498), a single-arm trial, treated 199 adults with relapsed or refractory IDH2-mutated AML at 100 mg daily. Complete remission was reached by 19% and complete remission with partial count recovery by a further 4%, for a combined rate of 23%, with a median response duration of 8.2 months; the median time to first response was 1.9 months. Among 157 patients who needed transfusions at the start, 34% became transfusion independent[1].
Development history
Explore Acute Myeloid Leukemia trials
Other Acute Myeloid Leukemia treatments
Common questions about Enasidenib
▸What is Enasidenib (Idhifa)?
A once-daily pill for adults whose AML has relapsed or never responded and carries a mutation in the IDH2 gene, found in roughly 1 in 10 people with AML. Approved August 1, 2017 as the first IDH2 inhibitor; it works by making blasts mature rather than by killing them outright.
▸How does Enasidenib work?
A mutated IDH2 enzyme makes large amounts of a chemical called 2-hydroxyglutarate, which blocks the steps that let a young blood cell mature. Enasidenib blocks the mutant enzyme (R140 and R172 forms), 2-hydroxyglutarate falls, and the blasts start to differentiate into normal-looking blood cells[1]. Because the leukemia cells mature rather than die, responses can take months, and the maturing cells can trigger differentiation syndrome.
▸What are the side effects of Enasidenib?
Idhifa carries a boxed warning for differentiation syndrome, which affected 14% of trial patients and can be fatal; signs include fever, breathlessness, lung infiltrates, fluid around the lungs or heart, rapid weight gain, swollen lymph nodes, bone pain and kidney or liver problems, and it is treated with steroids and close monitoring[1]. The other warning is harm to an unborn baby[1]. The most common side effects (20% or more) were nausea (50%), diarrhea (43%), vomiting (34%), raised bilirubin and decreased appetite (34%); tumor lysis syndrome occurred in 6% and noninfectious leukocytosis (a rise in white cells from the maturing blasts) in 12%[1].
▸How is Enasidenib taken?
The dose is 100 mg by mouth once a day, with or without food, swallowed whole, until the leukemia progresses or side effects become unacceptable. Because responses are slow, the label recommends at least 6 months of treatment if the disease is not progressing[1]. A missed or vomited dose is taken as soon as possible the same day.
▸Is Enasidenib FDA approved?
Yes, Enasidenib (Idhifa) is FDA approved (2017) for the treatment of Acute Myeloid Leukemia.
Sources and references
Every factual claim on this page is drawn from the public sources listed below. Click any reference to open the original document.
- Celgene Corporation via openFDA · 2025-01-16. Idhifa (enasidenib) tablets prescribing information. https://api.fda.gov/drug/label.json?search=set_id:%22a5b4cdf0-3fa8-4c6c-80f6-8d8a00e3a5b6%22
- FDA. Idhifa approval history. https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=209606